Home-based monitoring of pulmonary function in patients with duchenne muscular dystroph

Gunnar M. Buyse, Christian Rummey, Thomas Meier, Mika Leinonen, Thomas Voit, Craig M McDonald, Oscar H. Mayer

Research output: Contribution to journalArticle

3 Scopus citations

Abstract

Background: Loss of pulmonary function is a main cause of early morbidity and mortality in patients with Duchenne muscular dystrophy (DMD). Standard of care guidelines recommend regular assessment of pulmonary function by hospital-based spirometry to detect onset and monitor progression of pulmonary function decline. Objective: To assess the feasibility of home-based monitoring of pulmonary function by a hand-held device (HHD) in adolescent and adult patients with DMD over a period of 12 months. Methods: In the phase III randomized placebo-controlled DELOS trial in 10-18 year old DMD patients, peak expiratory flow (PEF) measurements were collected weekly at home by the patient (assisted by parent/caregiver) using a peak flow meter HHD. Adherence to the use of the HHD was assessed and 12-month changes in PEF as percent of predicted (PEF% p) for the idebenone (N = 31) and the placebo treatment groups (N = 33) from HHD-derived data were compared to results from hospital-based spirometry. Results: A total of 2689 individual HHD assessments were analysed. Overall adherence to the use of the HHD over the course of the 12-month study duration was good (75.9%, SD 21.5%) and PEF% p data obtained at the same day by HHD and standard spirometry correlated well (Spearman's rho 0.80; p < 0.001). Several analysis methods of HHD-derived data for PEF% p consistently demonstrate that idebenone treatment slowed the decline in PEF% p compared to placebo, which supports the statistically significant difference in favour of idebenone for PEF% p measured by standard spirometry. Conclusions: This study demonstrates that home-based monitoring of pulmonary function in adolescent patients with DMD using a HHD is feasible, provides reliable data compared to hospital-based spirometry and is therefore suitable for use in clinical practice and for clinical trials.

Original languageEnglish (US)
Pages (from-to)419-430
Number of pages12
JournalJournal of Neuromuscular Diseases
Volume5
Issue number4
DOIs
StatePublished - Jan 1 2018

Keywords

  • Duchenne muscular dystrophy
  • idebenone
  • peak expiratory flow
  • Pulmonary function
  • respiration

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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